Duchenne UK announces funding for a clinical trial to study the effects of Tamoxifen in Duchenne Muscular Dystrophy

Duchenne UK is delighted to announce that it is funding a clinical trial to assess the safety and efficacy of Tamoxifen as a potential treatment for Duchenne Muscular Dystrophy.

Duchenne UK is contributing £575,000 to the study and will fund one European trial site and two in the UK, along with the project manager for the study, based in Switzerland. Duchenne UK is working with the DMD Hub to support the trial sites in the UK for the Tamoxifen study. The UK sites will be Alder Hey Children’s Hospital in Liverpool and Leeds Children’s Hospital. Duchenne UK, along with Alex’s Wish and Joining Jack, are also providing funding to support clinical staff at Alder Hey and Leeds to deliver the trial.

Tamoxifen has been used to treat breast cancer since the 1980s. It is also used for hormonal disorders in pre-pubescent boys. Preliminary data in the DMD mouse model (Dorchies et al. 2013) demonstrated that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.

The trial is being jointly funded by Duchenne UK, E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy. The trial is due to begin in 2018.

Professor Dirk Fischer, from UKBB, the University Children’s Hospital in Basel, Switzerland in Switzerland who is running the study, said:

“Duchenne UK and the other patient organisations came in with funding at a very critical moment. Despite a positive evaluation and a recommendation by the E-Rare review process, this trial would not have been possible at all without the co-funding from Duchenne UK and the other patient organisations. I am extremely grateful for their ongoing support, which has included not only financial help, but also help negotiating with regulatory authorities and other involved parties."

"We are delighted to announce our funding and support for the Tamoxifen trial, and our support for the trial sites in the UK who will be running the study. Tamoxifen is a cheap and readily available medicine, with a good safety record in adults. We are keen to test this in patients with Duchenne Muscular Dystrophy. If this trial is successful, we could see a cheap, effective and readily available treatment for Duchenne Muscular Dystrophy in the very near future."

The trial marks an important milestone in Duchenne UK’s commitment to develop existing medicines as potential treatments for DMD, an approach known as repurposing. The advantage of this approach is that these medicines have already been tested for safety in humans and so their development time has the potential to be cut dramatically and they could offer a potentially quicker path to the clinic and approval.

Duchenne UK would like to thank its partner charities, Joining Jack, Alex’s Wish, For Felix and Jack’s Mission for supporting this study. We would also like to thank Caring for Connor, Brandon’s Fund, Team Felix, Smile with Shiv, Jack’s Mission, Help for Harry, Jayden's Army and Chasing Connor’s Cure, for their support of our ongoing work on repurposing.

- ENDS -

NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein

dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

Direct Debit – Duchenne Direct

Individual Donation – Donate

If you are a family or friend affected by DMD you can set up your own fund

Summit Therapeutics plc have announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. The data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies.

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Summit Therapeutics plc have announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. The data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies.

Yesterday, the new Care Considerations for Duchenne were published. We strongly encourage all parents, caregivers and patients to read these updates to the standards of care to ensure that everyone living with Duchenne has access to the best possible care

We’re pleased to share the latest coverage of Hercules. The launch of the project, with our seven industry partners, has received coverage in 10 publications. HERCULES is collaboration between Duchenne UK, and pharmaceutical companies to increase the chances of patients with DMD of accessing innovative treatments.

Duchenne UK is delighted to have been chosen by Marshall Wace as their charity of the year and to be awarded a donation of $100,000. The donation from Marshall Wace will fund 10 boys with Duchenne on our Tamoxifen Trial.

The DMD-Hub was created by Duchenne UK and leading neuromuscular clinicians. The DMD-Hub is addressing the lack of capacity in the UK for clinical trials in DMD. During the first year of operation the DMD Hub has successfully achieved, even surpassed, the immediate priorities identified through the Newcastle Plan.

We would like to congratulate the Joining Jack team for their recent victory! Joining Jack took a team to the Dubai 7's international rugby competition, and they came back champions! Alex Johnson, founder of Joining Jack and Jacks Mum, tells us more about Joining Jack's victory at the Dubai 7s.

It has been a busy year at Duchenne UK. Our mission is to fund and accelerate treatments and find a cure for Duchenne muscular dystrophy. We look at where we can add value to the drug development pathway, and how we can help to accelerate research that will benefit the whole community.

Today, December 14th, marks the publication of Janet Hoskin's book: 'A Guide to Duchenne Muscular Dystrophy: Information and Advice for Teachers and Parents'. We are offering free copies to families affected by DMD, please contact us to request a copy.

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Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

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